Drugs

FDA Approves New Safety Warning for Elevidys

FDA says it is approving new labeling submitted by Sarepta Therapeutics’ for its gene therapy Elevidys (delandistrogene moxeparvovec-rokl) that includes the addition of a Boxed Warning describing the risk of serious liver injury and acute liver failure, including fatal outcomes based on such reports in non-ambulatory patients treated with the product.

Elevidys is an AAVrh74 adeno-associated virus vector-based gene therapy approved for the treatment of Duchenne muscular dystrophy (DMD) in certain patients. In June, CBER issued a safety communication following two reports of fatal acute liver failure in non-ambulatory pediatric males with DMD after receiving Elevidys. In response, the manufacturer voluntarily paused distribution of Elevidys for use in non-ambulatory patients.

In both fatal cases, FDA says, patients developed markedly elevated liver enzymes and required hospitalization within two months of Elevidys infusion. An additional serious, non-fatal case of acute liver injury has involved complications such as mesenteric vein thrombosis, bowel ischemia and necrosis, and portal hypertension.

After a comprehensive evaluation of the available safety data, the agency has now approved the Boxed Warning and substantial labeling revisions for Elevidys, including:

Liver monitoring: Weekly liver function tests are advised for at least three months after treatment.
Prompt medical attention: Patients should contact their health care provider immediately if they experience yellowing of the skin or eyes, if they miss or vomit corticosteroid doses, or if the patient experiences a change in mental status.
Infection risk: Corticosteroid therapy may suppress immune function, increasing susceptibility to infections and serious complications including death.
Cardiac monitoring: Weekly testing for cardiac injury (troponin-I) is advised for one month following treatment.
Contraindications: Elevidys should not be used in patients with deletions involving DMD exons 8 and/or 9.
Limitations of Use: Elevidys is not recommended in patients with preexisting liver impairment, recent vaccinations, or recent/active infections.

Additionally, FDA says it is requiring Sarepta to conduct a postmarketing observational study to further assess the risk of serious liver injury. The study will enroll approximately 200 patients with DMD and follow them for at least 12 months after administration of Elevidys, with periodic liver function assessments.

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